Article

TALEN Mediated Gene Targeting for CF Gene Therapy

2018-11-19

Abstract excerpt

Cystic Fibrosis (CF) is an inherited monogenic disorder, amenable to gene based therapies. Because CF lung disease is currently the major cause of mortality and morbidity, and lung airway is readily accessible to gene delivery, the major CF gene therapy effort at present is directed to the lung. Although airway epithelial cells are renewed slowly, permanent gene correction through gene editing or targeting in airw...

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Literature Corpus work: ceb7ef88-0566-5ea3-91e3-6520f8421ee6
DOI: 10.20944/preprints201811.0444.v1

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